Phase 3
N=18
An Efficacy and Safety Study of Alirocumab in Children and Adolescents With Homozygous Familial Hypercholesterolemia
Hypercholesterolemia
Bottom Line
View on ClinicalTrials.gov: NCT03510715 ↗Enrolled (actual)
18
Serious AEs
5.6%
Results posted
Dec 2020
Primary outcome: Primary: Percent Change From Baseline in Low-Density Lipoprotein Cholesterol (LDL-C) at Week 12: Intent-to-Treat (ITT) Analysis — -4.1 percent change
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Alirocumab SAR236553 (REGN727) (Drug); Atorvastatin (Drug); Simvastatin (Drug); Fluvastatin (Drug); Pravastatin (Drug); Lovastatin (Drug); Rosuvastatin (Drug); Ezetimibe (Drug); Cholestyramine (Drug); Nicotinic acid (Drug); Fenofibrate (Drug); Omega-3 fatty acids (Drug)
- Age
- Pediatric · 8+ yrs
- Sex
- All
- Sponsor
- Sanofi
- Primary completion
- Feb 2020
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percent Change From Baseline in Low-Density Lipoprotein Cholesterol (LDL-C) at Week 12: Intent-to-Treat (ITT) Analysis |
-4.1 | — |
| SECONDARY Percent Change From Baseline in Low-Density Lipoprotein Cholesterol at Week 12: On-treatment Analysis |
-4.1 | — |
| SECONDARY Percent Change From Baseline in Low-Density Lipoprotein Cholesterol at Weeks 24 and 48: ITT Analysis/On-treatment Analysis |
-10.1; 4.2 | — |
| SECONDARY Percent Change From Baseline in Apolipoprotein (Apo) B at Weeks 12, 24 and 48: ITT Analysis/On-treatment Analysis |
-4.2; -11.8; 0.9 | — |
| SECONDARY Percent Change From Baseline in Non-High Density Lipoprotein Cholesterol (Non-HDL-C) at Weeks 12, 24 and 48 - ITT Analysis/On-treatment Analysis |
-3.9; -9.2; 5.7 | — |
| SECONDARY Percent Change From Baseline in Total Cholesterol (Total-C) at Weeks 12, 24 and 48: ITT Analysis/On-treatment Analysis |
-1.9; -6.3; 5.5 | — |
| SECONDARY Percent Change From Baseline in Lipoprotein a (Lp) (a) at Weeks 12, 24 and 48: ITT Analysis/On-treatment Analysis |
7.4; -5.2; -6.4 | — |
| SECONDARY Percent Change From Baseline in High Density Lipoprotein Cholesterol (HDL-C) at Weeks 12, 24 and 48: ITT Analysis/On-treatment Analysis |
13.0; 8.9; 10.1 | — |
| SECONDARY Percent Change From Baseline in Fasting Triglycerides (TG) at Weeks 12, 24 and 48: ITT Analysis/On-treatment Analysis |
2.8; 5.2; 10.0 | — |
| SECONDARY Percent Change From Baseline in Apolipoprotein A1 (Apo A1) at Weeks 12, 24 and 48: ITT Analysis/On-treatment Analysis |
11.3; 14.6; 11.3 | — |
| SECONDARY Percentage of Participants Reporting >=15 Percent (%) Reduction in LDL-C Level at Weeks 12, 24 and 48: ITT Analysis/On-treatment Analysis |
50.0; 50.0; 39.0 | — |
| SECONDARY Absolute Change From Baseline in LDL-C Level at Weeks 12, 24 and 48: ITT Analysis/On-treatment Analysis |
-33.4; -43.0; -15.0 | — |
| SECONDARY Number of Participants With Tanner Staging at Baseline, Weeks 12, 24 and 48 |
3; 0; 6; 9; 0; 0 | — |
Summary
Primary Objective:
To evaluate the efficacy of alirocumab (75 or 150 milligrams [mg] depending on body weight [BW]), administered every 2 weeks (Q2W), on low-density lipoprotein cholesterol (LDL-C) levels at Week 12 of treatment in children and adolescents with homozygous familial hypercholesterolemia (hoFH) of 8 to 17 years of age on top of background treatments.
Secondary Objectives:
* To evaluate the efficacy of alirocumab after 24 and 48 weeks of treatment on LDL-C levels.
* To evaluate the effects of alirocumab on other lipid parameters (eg, apolipoprotein B [Apo B], non-high density lipoprotein cholesterol [non-HDL-C], total cholesterol [Total-C], high density lipoprotein cholesterol [HDL-C], lipoprotein a [Lp (a)], triglycerides [TG], apolipoprotein A-1 [Apo A-1] levels) after 12, 24, and 48 weeks of treatment.
* To evaluate the safety and tolerability of alirocumab up to 48 weeks of treatment.
Eligibility Criteria
Inclusion criteria
- Participants genetically diagnosed with hoFH.
- Participants treated with optimal dose of statin +/- other lipid modifying therapies (LMTs), or non-statin LMTs if statin-intolerant at stable dose(s) for at least 4 weeks.
- A signed informed consent indicating parental permission with or without participants assent.
- For participants on apheresis, currently undergoing stable LDL apheresis therapy prior to the screening visit (Week -2) and had initiated apheresis treatment for at least 6 months.
Exclusion criteria
- Participants with LDL-C ) 350 mg/dL (3.95 mmol/L) at the screening visit.
- Severe renal impairment (i.e., estimated glomerular filtration rate 2 * upper limit of normal (ULN) at the screening visit.
- Creatine phosphokinase >3 * ULN at the screening visit.
The above information was not intended to contain all considerations relevant to a participants potential participation in a clinical trial.
Data sourced from ClinicalTrials.gov (NCT03510715). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.